ZA201701039B - An improved expression cassette for packaging and expression of variant factor viii for the treatment of hemostasis disorders - Google Patents

An improved expression cassette for packaging and expression of variant factor viii for the treatment of hemostasis disorders

Info

Publication number
ZA201701039B
ZA201701039B ZA2017/01039A ZA201701039A ZA201701039B ZA 201701039 B ZA201701039 B ZA 201701039B ZA 2017/01039 A ZA2017/01039 A ZA 2017/01039A ZA 201701039 A ZA201701039 A ZA 201701039A ZA 201701039 B ZA201701039 B ZA 201701039B
Authority
ZA
South Africa
Prior art keywords
expression
packaging
treatment
factor viii
variant factor
Prior art date
Application number
ZA2017/01039A
Inventor
Denise Sabatino
Katherine A High
Liron Elkouby
Original Assignee
Childrens Hospital Philadelphia
Denise Sabatino
Katherine A High
Liron Elkouby
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Childrens Hospital Philadelphia, Denise Sabatino, Katherine A High, Liron Elkouby filed Critical Childrens Hospital Philadelphia
Publication of ZA201701039B publication Critical patent/ZA201701039B/en

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/745—Blood coagulation or fibrinolysis factors
    • C07K14/755—Factors VIII, e.g. factor VIII C (AHF), factor VIII Ag (VWF)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P17/00—Drugs for dermatological disorders
    • A61P17/02—Drugs for dermatological disorders for treating wounds, ulcers, burns, scars, keloids, or the like
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/02—Antithrombotic agents; Anticoagulants; Platelet aggregation inhibitors
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/04—Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2840/00—Vectors comprising a special translation-regulating system
    • C12N2840/007—Vectors comprising a special translation-regulating system cell or tissue specific

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Epidemiology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Hematology (AREA)
  • Biophysics (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Diabetes (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Dermatology (AREA)
  • Cardiology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
ZA2017/01039A 2014-08-13 2017-02-10 An improved expression cassette for packaging and expression of variant factor viii for the treatment of hemostasis disorders ZA201701039B (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201462036936P 2014-08-13 2014-08-13
PCT/US2015/045142 WO2016025764A2 (en) 2014-08-13 2015-08-13 An improved expression cassette for packaging and expression of variant factor viii for the treatment of hemostasis disorders

Publications (1)

Publication Number Publication Date
ZA201701039B true ZA201701039B (en) 2021-08-25

Family

ID=55304765

Family Applications (1)

Application Number Title Priority Date Filing Date
ZA2017/01039A ZA201701039B (en) 2014-08-13 2017-02-10 An improved expression cassette for packaging and expression of variant factor viii for the treatment of hemostasis disorders

Country Status (12)

Country Link
US (1) US11014975B2 (en)
EP (1) EP3180022B1 (en)
JP (2) JP2018506261A (en)
KR (1) KR102665348B1 (en)
CN (1) CN107427557B (en)
AU (1) AU2015301598B2 (en)
BR (1) BR112017002781A2 (en)
CA (2) CA3237630A1 (en)
SA (1) SA517380883B1 (en)
SG (1) SG11201701033QA (en)
WO (1) WO2016025764A2 (en)
ZA (1) ZA201701039B (en)

Families Citing this family (17)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US10058624B2 (en) * 2015-04-16 2018-08-28 Emory University Recombinant promoters and vectors for protein expression in liver and use thereof
JP6929230B2 (en) * 2015-05-14 2021-09-01 セント ジュード チルドレンズ リサーチ ホスピタル インコーポレイテッド Nucleic acid molecules containing spacers and methods of their use
US11230720B2 (en) 2015-10-14 2022-01-25 Audentes Therapeutics, Inc. Nucleic acid molecules containing spacers and methods of use thereof
PE20231949A1 (en) * 2015-10-30 2023-12-05 Spark Therapeutics Inc VARIANTS OF FACTOR VIII REDUCED WITH CpG, COMPOSITIONS AND METHODS AND USES FOR THE TREATMENT OF HEMOSTASY DISORDERS
EA036944B1 (en) 2015-11-13 2021-01-19 Баксалта Инкорпорейтед Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a
IL281038B2 (en) 2015-11-13 2024-02-01 Baxalta Inc Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a
CN111163796A (en) * 2017-08-01 2020-05-15 星火治疗有限公司 Factor VIII (FVIII) gene therapy methods
BR112020022164A2 (en) 2018-05-18 2021-02-02 Bioverativ Therapeutics Inc. methods of treating hemophilia a
CN108795986A (en) * 2018-05-31 2018-11-13 深圳市免疫基因治疗研究院 A kind of haemophilia A slow virus carrier, slow virus and its preparation method and application
MX2021000582A (en) 2018-07-16 2021-04-12 Baxalta Inc Gene therapy of hemophilia a using viral vectors encoding recombinant fviii variants with increased expression.
CN113614104A (en) 2019-01-16 2021-11-05 百深公司 Viral vectors encoding recombinant FVIII variants with increased expression for gene therapy of hemophilia A
CN111218446B (en) * 2019-12-25 2023-03-28 劲帆生物医药科技（武汉）有限公司 Liver specificity promoter and application thereof
WO2022241041A2 (en) * 2021-05-11 2022-11-17 City Of Hope Adeno-associated virus compositions and methods of use thereof
WO2024007978A1 (en) * 2022-07-07 2024-01-11 深圳新诺微环生物科技有限公司 Linker peptide, fviii protein containing linker peptide or variant thereof, and use thereof
CN115948408A (en) * 2022-09-23 2023-04-11 上海信致医药科技有限公司 Improved human coagulation factor VIII gene expression cassette and application thereof
CN115873099B (en) * 2022-12-30 2023-10-20 福因医药科技(武汉)有限公司 Modified recombinant blood coagulation factor VIII and application thereof
CN116715752A (en) * 2023-06-16 2023-09-08 苏州诺洁贝生物技术有限公司 Coagulation factor FVIII protein variants, expression vectors and uses

Family Cites Families (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6114148C1 (en) 1996-09-20 2012-05-01 Gen Hospital Corp High level expression of proteins
US6924365B1 (en) * 1998-09-29 2005-08-02 Transkaryotic Therapies, Inc. Optimized messenger RNA
US6221349B1 (en) * 1998-10-20 2001-04-24 Avigen, Inc. Adeno-associated vectors for expression of factor VIII by target cells
ES2426245T3 (en) * 2005-04-07 2013-10-22 The Trustees Of The University Of Pennsylvania Method to increase the functionality of an AAV vector
CN104087591A (en) * 2006-06-19 2014-10-08 阿斯克肋匹奥生物制药公司 Modified factor viii and factor ix genes and vectors for gene therapy
US20120028900A1 (en) * 2006-06-30 2012-02-02 Kaufman Randal J Method of producing factor viii proteins by recombinant methods
CN101597616A (en) * 2008-06-04 2009-12-09 上海同科生物科技有限公司 A kind of method that improves expression quantity of gene recombinant human coagulation factor 8
AU2013202564B2 (en) * 2008-06-24 2015-09-17 Csl Behring Gmbh Factor VIII, von Willebrand factor or complexes thereof with prolonged in vivo half-life
GB0911870D0 (en) * 2009-07-08 2009-08-19 Ucl Business Plc Optimised coding sequence and promoter
ES2717902T5 (en) * 2009-10-02 2022-11-29 Childrens Hospital Philadelphia Compositions and methods for improving the function of coagulation factor VIII
WO2014008480A2 (en) * 2012-07-06 2014-01-09 Biogen Idec Ma Inc. Cell line expressing single chain factor viii polypeptides and uses thereof
JP6454643B2 (en) * 2012-10-26 2019-01-16 フリーイェ・ユニヴェルシテイト・ブリュッセルＶｒｉｊｅ Ｕｎｉｖｅｒｓｉｅｉｔ Ｂｒｕｓｓｅｌ Vectors and methods and uses for liver-directed gene therapy for hemophilia
SI3889173T1 (en) * 2013-02-15 2023-11-30 Bioverativ Therapeutics Inc. Optimized factor viii gene

Also Published As

Publication number Publication date
BR112017002781A2 (en) 2017-12-19
CA2958141C (en) 2024-06-11
CA2958141A1 (en) 2016-02-18
KR102665348B1 (en) 2024-05-16
AU2015301598A1 (en) 2017-03-23
SG11201701033QA (en) 2017-03-30
CN107427557A (en) 2017-12-01
KR20170084012A (en) 2017-07-19
WO2016025764A2 (en) 2016-02-18
WO2016025764A3 (en) 2016-04-07
JP7237903B2 (en) 2023-03-13
EP3180022A4 (en) 2019-02-20
JP2018506261A (en) 2018-03-08
US11014975B2 (en) 2021-05-25
CN107427557B (en) 2022-01-04
SA517380883B1 (en) 2021-12-18
US20170233456A1 (en) 2017-08-17
JP2021003120A (en) 2021-01-14
EP3180022A2 (en) 2017-06-21
AU2015301598B2 (en) 2020-07-16
EP3180022B1 (en) 2024-07-03
CA3237630A1 (en) 2016-02-18

Similar Documents

Publication Publication Date Title
ZA201701039B (en) An improved expression cassette for packaging and expression of variant factor viii for the treatment of hemostasis disorders
HK1243901A1 (en) Devices and methods for ablation of the skin
EP3212191A4 (en) Treatment for depression and depressive disorders
EP3995086C0 (en) Medical device for modification of left atrial appendage
EP3164080A4 (en) Methods and devices for surgical access
EP3151797A4 (en) Methods and devices for treating the skin
EP3193795A4 (en) Devices and methods for the removal of lenticular tissue
EP3256218A4 (en) Kdm1a inhibitors for the treatment of disease
IL248538A0 (en) Adenoassociated virus vectors for the treatment of lysosomal storage disorders
EP3171820A4 (en) Implantable devices and techniques for oncoplastic surgery
HK1244485A1 (en) Combinations for the treatment of neuroblastoma
PL3247459T3 (en) Device for the ultrasound treatment of open wounds
HK1232220A1 (en) Derivatives of 5-benzylisoquinoline for the treatment of cardiovascular diseases 5-
IL249502A0 (en) Novel therapeutic uses of benzylideneguanidine derivatives for the treatment of proteopathies
IL248210A0 (en) Methods and compositions for the treatment of vascular malformation
EP3258930A4 (en) Oxabicycloheptanes and oxabicycloheptenes for the treatment of depressive and stress disorders
IL251736B (en) Methods and compositions for the treatment of radiation-related disorders
GB201423272D0 (en) The combination for anti-wrinkle and soothing the skin
PL3023210T3 (en) Method for the treatment of distributable goods
EP3145525A4 (en) Use of microperoxidases for the treatment of carboxyhemoglobinemia
ZA201700008B (en) Device for medical thermotherapy
IL248494A0 (en) Methods and compositions for treatment of lipid storage disorders
EP3200795A4 (en) Use of agents for treating fat-related disorders
EP3222281A4 (en) Preventive and therapeutic agent for celiac disease
HUE052098T2 (en) Prohemostatic proteins for the treatment of bleeding